Literature DB >> 29414930

Gene therapies for hemophilia hit the mark in clinical trials.

Adrian K Pickar1, Charles A Gersbach1.   

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Year:  2018        PMID: 29414930     DOI: 10.1038/nm.4492

Source DB:  PubMed          Journal:  Nat Med        ISSN: 1078-8956            Impact factor:   53.440


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  12 in total

1.  In vivo genome editing of the albumin locus as a platform for protein replacement therapy.

Authors:  Rajiv Sharma; Xavier M Anguela; Yannick Doyon; Thomas Wechsler; Russell C DeKelver; Scott Sproul; David E Paschon; Jeffrey C Miller; Robert J Davidson; David Shivak; Shangzhen Zhou; Julianne Rieders; Philip D Gregory; Michael C Holmes; Edward J Rebar; Katherine A High
Journal:  Blood       Date:  2015-08-21       Impact factor: 22.113

2.  The prospect for designed genetic change.

Authors:  R L Sinsheimer
Journal:  Am Sci       Date:  1969       Impact factor: 0.548

3.  AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

Authors:  Savita Rangarajan; Liron Walsh; Will Lester; David Perry; Bella Madan; Michael Laffan; Hua Yu; Christian Vettermann; Glenn F Pierce; Wing Y Wong; K John Pasi
Journal:  N Engl J Med       Date:  2017-12-09       Impact factor: 91.245

4.  Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors:  Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal:  N Engl J Med       Date:  2017-11-02       Impact factor: 91.245

5.  Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.

Authors:  Narendra Maheshri; James T Koerber; Brian K Kaspar; David V Schaffer
Journal:  Nat Biotechnol       Date:  2006-01-22       Impact factor: 54.908

6.  Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

Authors:  Lindsey A George; Spencer K Sullivan; Adam Giermasz; John E J Rasko; Benjamin J Samelson-Jones; Jonathan Ducore; Adam Cuker; Lisa M Sullivan; Suvankar Majumdar; Jerome Teitel; Catherine E McGuinn; Margaret V Ragni; Alvin Y Luk; Daniel Hui; J Fraser Wright; Yifeng Chen; Yun Liu; Katie Wachtel; Angela Winters; Stefan Tiefenbacher; Valder R Arruda; Johannes C M van der Loo; Olga Zelenaia; Daniel Takefman; Marcus E Carr; Linda B Couto; Xavier M Anguela; Katherine A High
Journal:  N Engl J Med       Date:  2017-12-07       Impact factor: 91.245

7.  Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

Authors:  Jenny McIntosh; Peter J Lenting; Cecilia Rosales; Doyoung Lee; Samira Rabbanian; Deepak Raj; Nishil Patel; Edward G D Tuddenham; Olivier D Christophe; John H McVey; Simon Waddington; Arthur W Nienhuis; John T Gray; Paolo Fagone; Federico Mingozzi; Shang-Zhen Zhou; Katherine A High; Maria Cancio; Catherine Y C Ng; Junfang Zhou; Christopher L Morton; Andrew M Davidoff; Amit C Nathwani
Journal:  Blood       Date:  2013-02-20       Impact factor: 22.113

8.  Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

Authors:  Catherine S Manno; Glenn F Pierce; Valder R Arruda; Bertil Glader; Margaret Ragni; John J Rasko; John Rasko; Margareth C Ozelo; Keith Hoots; Philip Blatt; Barbara Konkle; Michael Dake; Robin Kaye; Mahmood Razavi; Albert Zajko; James Zehnder; Pradip K Rustagi; Hiroyuki Nakai; Amy Chew; Debra Leonard; J Fraser Wright; Ruth R Lessard; Jürg M Sommer; Michael Tigges; Denise Sabatino; Alvin Luk; Haiyan Jiang; Federico Mingozzi; Linda Couto; Hildegund C Ertl; Katherine A High; Mark A Kay
Journal:  Nat Med       Date:  2006-02-12       Impact factor: 53.440

9.  Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs.

Authors:  Haiyan Jiang; David Lillicrap; Susannah Patarroyo-White; Tongyao Liu; Xiaobing Qian; Ciaran D Scallan; Sandra Powell; Tracey Keller; Morag McMurray; Andrea Labelle; Dea Nagy; Joseph A Vargas; Shangzhen Zhou; Linda B Couto; Glenn F Pierce
Journal:  Blood       Date:  2006-03-07       Impact factor: 22.113

10.  Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Authors:  Amit C Nathwani; Ulreke M Reiss; Edward G D Tuddenham; Cecilia Rosales; Pratima Chowdary; Jenny McIntosh; Marco Della Peruta; Elsa Lheriteau; Nishal Patel; Deepak Raj; Anne Riddell; Jun Pie; Savita Rangarajan; David Bevan; Michael Recht; Yu-Min Shen; Kathleen G Halka; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; James Allay; Mark A Kay; Catherine Y C Ng; Junfang Zhou; Maria Cancio; Christopher L Morton; John T Gray; Deokumar Srivastava; Arthur W Nienhuis; Andrew M Davidoff
Journal:  N Engl J Med       Date:  2014-11-20       Impact factor: 176.079

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  2 in total

Review 1.  Towards translational optogenetics.

Authors:  Akshaya Bansal; Swati Shikha; Yong Zhang
Journal:  Nat Biomed Eng       Date:  2022-01-13       Impact factor: 29.234

2.  A Treg-Selective IL-2 Mutein Prevents the Formation of Factor VIII Inhibitors in Hemophilia Mice Treated With Factor VIII Gene Therapy.

Authors:  Alex C Chen; Xiaohe Cai; Chong Li; Liliane Khoryati; Marc A Gavin; Carol H Miao
Journal:  Front Immunol       Date:  2020-04-28       Impact factor: 7.561

  2 in total

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